On Success of One-Patient Study, TransDerm Aims to Advance siRNA Drug for Rare Skin Disorder | GenomeWeb

Data from a one-patient study of an investigational siRNA-based treatment for the rare skin disorder pachyonychia congenita indicates the drug is safe and potentially effective, an official from the company developing the therapy told RNAi News last week.

However, because the route of administration, intradermal injections, proved more painful than anticipated, a planned follow-up trial will likely use an investigational cream-based delivery vehicle, TransDerm Founder and CEO Roger Kaspar said.

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